The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Oct. 14, 2025
Filed:
Mar. 06, 2020
Academisch Ziekenhuis Leiden (H.o.d.n. Lumc), Leiden, NL;
Frank J. T. Staal, Leiden, NL;
Karin Pike-Overzet, Leiden, NL;
Laura Garcia-Perez, Leiden, NL;
Marja C. J. A. Van Eggermond, Leiden, NL;
Arjan C. Lankester, Leiden, NL;
ACADEMISCH ZIEKENHUIS LEIDEN (H.O.D.N. LUMC), Leiden, NL;
Abstract
The present invention provides novel expression cassettes, retroviral plasmids, vectors, virions, compositions and recombinant cells comprising a promoter operably linked to a codon optimised recombination activating (RAG1) transgene. These novel expression cassettes, retroviral plasmids, vectors, virions, compositions and recombinant cells are useful in the treatment of diseases caused by complete or partial loss-of-function of the protein encoded by the rag-1 gene, such as RAG-deficient severe combined immunodeficiency (RAG1-SCID), Omenn Syndrome (OS), atypical-SCID or combined immunodeficiency (CID). Corresponding methods of treatment are also provided.